Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia

Inclusion criteria

• Diagnosis of genetically confirmed Friedreich's ataxia
• Visual acuity at baseline more than 15 letters on EDTRS at four meters
• FARS score of 20 to 90
• Agreement to use contraception if within reproductive years (see specifics in section D1, page 21)
• Hormone replacement therapy, if used, must remain stable for the duration of the study
• Willingness and ability to comply with study procedures
• Willingness and ability to arrive at study site day prior to evaluations
• Abstention from use of dietary supplements and non-prescribed medications at least 30 days prior to initiation of treatment and for the duration of the study. This would specifically include idebenone, Coenzyme Q10 and vitamin E
• Abstention from use of other investigative or non-approved drugs within 30 days of enrollment and for the duration of the study

Exclusion criteria

• Allergy to EPI-743 or sesame oil or nuts
• Clinically significant bleeding condition or abnormal PT/PTT INR (INR > two; PTT > two-times normal)
• Liver insufficiency with LFTs greater than three-times upper normal limit at screening
• Renal insufficiency with creatinine > 1.5 at screening
• Fat malabsorption syndromes
• Any other respiratory chain diseases of the mitochondria or inborn errors of metabolism
• Any other ophthalmologic conditions
• History of alcohol or drug abuse
• Clinically significant cardiomyopathy with ejection fraction < 40 percent at screening
• Clinically significant arrhythmia within past two years requiring treatment
• Anticoagulant therapy within 30 days of enrollment.